Mr. Brownback (for
himself, Mr. Brown of Ohio, and
Mr. Franken) introduced the following
bill; which was read twice and referred to the
Committee on Health, Education, Labor,
and Pensions

A BILL

To amend the Federal Food, Drug, and Cosmetic Act to
improve the priority review voucher incentive program relating to tropical and
rare pediatric diseases.

1.

Short
title; references

(a)

Short
title

This Act may be cited
as the Creating Hope Act of
2010.

(b)

References

Wherever
in this Act an amendment is expressed in terms of an amendment to a section or
other provision, the reference shall be considered to be made to a section or
other provision of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 301 et
seq.).

by redesignating
paragraphs (3) and (4) as paragraphs (6) and (7), respectively;

(2)

by redesignating
paragraphs (1) and (2) as paragraphs (2) and (3), respectively;

(3)

by inserting
after In this section:, the following:

(1)

Innovative
treatment

The term innovative treatment
means—

(A)

a human drug that
is the subject of an application submitted under section 505(b)(1), if that
drug contains no active ingredient (including any ester or salt of the active
ingredient) that has been previously approved in any other application under
section 505(b)(1), 505(b)(2), or 505(j) or section 351 of the Public Health
Service Act; or

(B)

a biological
product that is the subject of an application submitted under section 351(a) of
the Public Health Service Act, if that biological product—

(i)

does not have the
same structure as a biological product that has been previously licensed in any
other application under subsection (a) or (k) of section 351 of the Public
Health Service Act or approved under section 505 of this Act; and

(ii)

is not
biosimilar, within the meaning of section 351(i) of the Public Health Service
Act, to a biological product that has been previously licensed in any other
application under subsection (a) or (k) of section 351 of the Public Health
Service Act or approved under section 505 of this
Act.

;

(4)

in paragraph (3),
as so redesignated, by inserting or rare pediatric disease product
application after tropical disease product application
each place that phrase appears;

(5)

by inserting
after paragraph (3) the following:

(4)

Rare pediatric
disease

The term rare pediatric disease means a
disease that meets each of the following criteria:

(A)

The disease is
recognized in the medical community as affecting a pediatric population.

(B)

The disease is a
rare disease or condition, within the meaning of section 526.

that relies on
clinical data derived from studies examining a pediatric population and dosages
of the drug intended for that population; and

(E)

that does not
seek approval for an adult indication in the original rare pediatric disease
product
application.

;

(6)

in paragraph (6),
as so redesignated—

(A)

by redesignating
subparagraph (Q) as subparagraph (R); and

(B)

by inserting
after subparagraph (P) the following:

(Q)

Chagas
Disease.

;
and

(7)

by amending
paragraph (7), as so redesignated, to read as follows:

(7)

Tropical
disease product application

The term tropical disease
product application means a human drug application, as defined in
section 735(1)—

(A)

for prevention or
treatment of a tropical disease;

(B)

that the
Secretary deems eligible for priority review;

(C)

that is for an
innovative treatment; and

(D)

that is for a
drug that has not been approved for commercial marketing for any tropical
disease indication by a government authority outside of the United States for
more than 24 months before the tropical disease product application is
submitted.

.

(c)

Rules regarding
use and transfer of priority review vouchers

Section 524(b) (21
U.S.C. 360n(b)) is amended—

(1)

in paragraph (1),
by inserting or rare pediatric disease product application after
tropical disease product application each place that phrase
appears;

(2)

by amending
paragraph (2) to read as follows:

(2)

Transferability

(A)

In
general

The sponsor of a tropical disease product application or
rare pediatric disease product application that receives a priority review
voucher under this section may transfer (including by sale) the entitlement to
such voucher. There is no limit on the number of times a priority review
voucher may be transferred before such voucher is used.

(B)

Conditions of
transfer

If a sponsor transfers a priority review voucher after
such sponsor has provided notification to the Secretary under paragraph (4)(A)
of the intent of such sponsor to use the voucher, the transfer shall be subject
to the provisions of subparagraphs (B) and (C) of paragraph (4).

(C)

Notification of
transfer

The person to whom a voucher is transferred under
paragraph (4)(B)(i) shall notify the Secretary of such change in ownership of
the voucher not later than 30 days after such
transfer.

;

(3)

by amending
paragraph (3) to read as follows:

(3)

Limitation for
prior applications

(A)

Tropical
disease product applications

A sponsor of a tropical disease
product application may not receive a priority review voucher under this
section if the tropical disease product application was submitted to the
Secretary prior to September 27, 2007.

(B)

Rare pediatric
disease product applications

A sponsor of a rare pediatric
disease product application may not receive a priority review voucher under
this section if the rare pediatric disease product application was submitted to
the Secretary prior to the date that is 90 days after the date of enactment of
the Creating Hope Act of 2010.

;
and

(4)

by amending
paragraph (4) to read as follows:

(4)

Notification

(A)

Timing

At
least 90 days before the date on which a human drug application for which the
sponsor intends to use a priority review voucher is submitted, the sponsor of
such human drug application shall notify the Secretary of the intent of such
sponsor to submit the human drug application.

(B)

Transfer of
voucher after notification

(i)

In
general

The sponsor of a human drug application that provides
notification of the intent of such sponsor to use the voucher for the human
drug application may transfer the voucher within 1 year after such notification
is provided, if such sponsor has not yet submitted the human drug application
described in the notification.

(ii)

Exception

The
person to whom a voucher is transferred under clause (i) (referred to in this
paragraph as the transferee) shall give notification of the intent
of such transferee to use the voucher in accordance with this subsection,
unless—

(I)

the transferee
uses the voucher for a human drug application featuring the same indications as
the human drug application described in the transferor’s notification;
and

(II)

the transferee
notifies the Secretary within 30 days of the transfer of the intent of such
transferee to use the voucher for such purpose.

(iii)

Internal
transfer

If the sponsor transfers a voucher internally for use
with a drug application that includes one or more indications that were not
included in the drug application that was the subject of the notification of
such sponsor, the sponsor shall notify the Secretary of the transfer in
accordance with this subsection.

(C)

Fee due upon
notification; credit for transferred voucher

(i)

Due upon
notification

The notification under this subsection shall be a
legally binding commitment to pay for the user fee to be assessed in accordance
with this section. Such fee shall be payable by the sponsor upon the submission
by such sponsor of such notification.

(ii)

Credit

If
a sponsor pays a user fee upon providing notification of the intent of such
sponsor to use a priority review voucher, but later transfers the voucher for
which such sponsor gave notification, the Secretary shall credit the user fees
paid to the next human drug application for which a sponsor provides
notification of the intent of such sponsor to use the same transferred
voucher.

(iii)

Difference in
fee

The Secretary may require a sponsor using a transferred
voucher to pay the difference between the credit associated with the
transferred voucher and the user fee prevailing at the time the sponsor submits
notification of the intent of such sponsor to use the transferred voucher. This
provision does not apply in cases where a transferee is exempted from
submitting notification under this
paragraph.

.

(d)

Payment

Section
524(c)(4) (21 U.S.C. 360n(c)(4)) is amended—

(1)

in subparagraph
(A), by striking submission of a human drug application under section
505(b)(1) or section 351 of the Public Health Services Act for which the
priority review voucher is used. and inserting notification by a
sponsor of the intent of such sponsor to use the voucher, as specified in
subsection (b)(4)(A). All other user fees associated with the human drug
application shall be due as required by the Secretary or under applicable
law.; and

(2)

in subparagraph
(C), by striking the period at the end and inserting , except as
specified in subsection (b)(4)(C)..

(e)

Designation
process; product implementation requirement

Section 524 (21
U.S.C. 360n) is amended by adding at the end the following new
subsections:

(e)

Designation
process

(1)

Designation of
rare pediatric diseases

(A)

In
general

Upon the request of the manufacturer or the sponsor of a
new drug, the Secretary may designate that the new drug is for a rare pediatric
disease. Such a request for designation, if sought, shall be made when
requesting designation of orphan disease status under section 526 or fast-track
designation under section 506. Requesting designation of rare pediatric disease
status under this paragraph is not a prerequisite to receiving a priority
review voucher.

(B)

Determination
by Secretary

Not later than 60 days after a request is submitted
under subparagraph (A), the Secretary shall determine whether the disease or
condition that is the subject of such request is a rare pediatric
disease.

(2)

Designation of
innovative treatments

(A)

In
general

Upon the request of the manufacturer or the sponsor of a
new drug, the Secretary may designate that a new drug is an innovative
treatment. Such a request for designation, if sought, shall be made when
requesting fast-track designation under section 506. Requesting designation
that a new drug is an innovative treatment is not a prerequisite to receiving a
priority review voucher.

(B)

Determination
by Secretary

Not later than 60 days after a request is submitted
under subparagraph (A), the Secretary shall determine whether the new drug that
is the subject of such request is an innovative treatment.

(f)

Product
implementation for rare pediatric disease products

(1)

In
general

The Secretary shall deem a rare pediatric disease product
application incomplete if such application does not contain a description of
the plan of the sponsor of such application to market the product in the United
States.

(2)

Good faith
intent to market

(A)

Good faith
intent required

The Secretary may refuse to issue a priority
review voucher upon the approval of a rare pediatric disease product
application if the Secretary finds that the sponsor of such application lacks a
good faith intention to produce and distribute the product. The Secretary may
consider any fact relevant to this determination, including the history of such
sponsor of producing rare pediatric disease products for which such sponsor
received a priority review voucher, orphan drugs for which the sponsor received
exclusivity under section 527, or pediatric drugs for which the sponsor
received an additional 6 months of exclusivity under section 505A.

(B)

Presumption

The
sponsor may establish a presumption of good faith by demonstrating that such
sponsor has allocated sufficient resources or otherwise arranged for the
production of the rare pediatric disease product in a manner sufficient to meet
the expected demand for the product during the 5-year period following approval
of the application.

(3)

Production
report

(A)

Report
required

The sponsor of an approved rare pediatric disease
product shall submit a report to the Secretary not later than 5 years after the
approval of the applicable rare pediatric disease product application. Such
report shall provide the following information, with respect to each of the
first 4 years after approval of such product:

(i)

The estimated
population in the United States suffering from the rare pediatric
disease.

(ii)

The estimated
demand in the United States for such rare pediatric disease product.

(iii)

The actual
amount of such rare pediatric disease product distributed in the United
States.

(B)

Publication
upon failure to demonstrate good faith effort to market

The
Secretary may publish the results of a report submitted under subparagraph (A)
in the Federal Register if the Secretary finds that the sponsor that submitted
such report has not made a good faith effort to meet the demand in the United
States for the product that is the subject of such report during each of the
first 4 years after approval of such product.

(g)

Production
report for tropical disease products

(1)

Report
required

The sponsor of an approved tropical disease product
shall submit a report to the Secretary not later than 5 years after the
approval of the applicable rare tropical disease product application. Such
report shall provide the following information, with respect to each of the
first 4 years after approval of such product:

(A)

The estimated
global population suffering from the tropical disease.

(B)

The estimated
global demand for such tropical disease product.

(C)

The actual amount
of such tropical disease product distributed globally.

(2)

Publication
upon failure to demonstrate good faith effort to market

The
Secretary may publish the results of a report submitted under paragraph (1) in
the Federal Register if the Secretary finds that the sponsor that submitted
such report has not made a good faith effort to meet the global demand for the
product that is the subject of such report during each of the first 4 years
after approval of such product.

(h)

Notice of
issuance and use of voucher

The Secretary shall publish a notice
in the Federal Register and on the Web site of the Food and Drug Administration
not later than 30 days after the occurrence of each of the following:

(1)

The Secretary
issues a priority review voucher under this section.

(2)

A sponsor submits
a human drug application for which such sponsor uses a priority review
voucher.

(i)

Eligibility for
other programs

A sponsor who seeks a priority review voucher
under this section may participate in any other incentive program, including
the programs the Secretary has implemented under this Act, if the sponsor meets
the applicable criteria of such other incentive program.

(j)

Relation to
other provisions

This provisions of this section shall
supplement, not supplant, any other provisions of this Act or the Public Health
Service Act that encourage the development of drugs for tropical diseases and
rare pediatric
diseases.